Artificially Induced Pluripotent Stem Cell-Derived Whole-Brain Organoid for Modelling the Pathophysiology of Metachromatic Leukodystrophy and Drug Repurposing

Metachromatic leukodystrophy (MLD) is a rare neurodegenerative disease that results from a deficiency of the lysosomal enzyme arylsulfatase A (ARSA). Worldwide, there are between one in 40,000 and one in 160,000 people living with the disease. While there are currently no effective treatments for ML...

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Detalles Bibliográficos
Autores principales: Esmail, Sally, Danter, Wayne R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8073899/
https://www.ncbi.nlm.nih.gov/pubmed/33923989
http://dx.doi.org/10.3390/biomedicines9040440

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8073899/
https://www.ncbi.nlm.nih.gov/pubmed/33923989
http://dx.doi.org/10.3390/biomedicines9040440

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